Treatment of the X-linked lymphoproliferative, Griscelli and Chédiak-Higashi syndromes by HLH directed therapy.
BACKGROUND
Griscelli syndrome type 2 (GS2), the X-linked lymphoproliferative (XLP) and the Chédiak-Higashi (CHS) syndromes are diseases that all may develop hemophagocytic syndromes. We wanted to investigate whether the treatment protocols for hemophagocytic lymphohistiocytosis (HLH) can also be used for these syndromes.


METHODS
In the HLH-94/HLH-2004 treatment study registries, we evaluated all patients with GS2 (n = 5), XLP (n = 2) or CHS (n = 2) treated between 1994 and 2004.


RESULTS
All patients responded to the therapy, and all are alive but one (suffering from CHS), with a mean follow-up of 5.6 years. All GS2, one XLP and one CHS patient underwent hematopoietic stem cell transplant. Mean follow-up post transplant was 6.0 years. Six of the seven transplanted children achieved non-active disease status at the time for SCT. Neurological sequelae were reported in all, except for the XLP patients.


CONCLUSIONS
Our results indicate that HLH treatment can be an effective first line treatment to induce remission in patients with GS2, XLP and CHS that have developed a hemophagocytic syndrome. We suggest that these patients should be included as a separate cohort in the international HLH study.
Trottestam
Helena
H
Childhood Cancer Research Unit, Karolinska University Hospital, Department of Woman and Child Health, Karolinska Institutet, Stockholm, Sweden. Helena.Trottestam@ki.se
Beutel
Karin
K
Meeths
Marie
M
Carlsen
Niels
N
Heilmann
Carsten
C
Pasić
Srdjan
S
Webb
David
D
Hasle
Henrik
H
Henter
Jan-Inge
JI
D000293:Adolescent; D002609:Chediak-Higashi Syndrome; D002648:Child; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D051359:Lymphohistiocytosis, Hemophagocytic / Q000628:therapy*; D008232:Lymphoproliferative Disorders / Q000628:therapy*; D008297:Male; D012042:Registries; D012074:Remission Induction; D012189:Retrospective Studies; D015996:Survival Rate; D016896:Treatment Outcome